The CHAMPIONS clinical research study is looking for people 18 and over with attenuated MPS II to test SB-913, a type investigational gene therapy called genome editing. (“Investigational” means it isn’t approved by the FDA for general use as a medicine.)

SB-913 consists of a substance (called a vector or carrier) that carries a healthy IDS enzyme for processing large molecules in the body. Study participants will receive a single infusion of SB-913 into a vein. The gene that will create the working IDS enzyme will then be carried through the bloodstream to the liver.

Doctors are studying SB-913 to see if it’s safe and tolerable, and learn more about its effectiveness at enabling your body to process large molecules on its own.

Yes. Study participants will be assigned into 1 of 3 treatment groups. Each treatment group will receive a different dose (low, medium, or high) of SB-913. This study is open-label, which means that you and your doctor will know which dose of SB-913 you received.

Will study participation cost me anything?

No. All study medication and study-related care will be at no cost to you. You’ll also receive reimbursement for travel. Transportation assistance may be available.

How long would study participation last?

After you receive the infusion of SB-913, your study staff will follow your health for up to 3 years. You’ll make regular study appointments with the study staff, and the staff will work with you to find convenient times for these visits. At these visits, the study staff will check your health, including taking blood, urine, saliva, stool, and semen samples to study the effects of the investigational therapy.

If for some reason you need to leave the study, you may do so at any time, for any reason.